- A single ABBV-RGX-314 gene therapy treatment has the
potential to become a new standard-of-care option among anti-VEGF
treatments by sustaining vision health long term and overcoming the
clinical challenges of managing wet AMD due to the treatment burden
of chronic anti-VEGF injections
- ABBV-RGX-314 continues to be well tolerated in over 100
patients from three dose levels with no drug-related serious
adverse events
- ABBV-RGX-314 in over 50 patients at third dose level
demonstrated highest reduction in treatment burden:
- 80% reduction in annualized injection rate
- 50% injection-free
- Zero cases of intraocular inflammation observed in patients
that received short-course prophylactic topical steroid eye
drops
- Conference call Tuesday, January
16 at 4:30 p.m. EST
ROCKVILLE, Md., Jan. 16,
2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX)
today announced positive interim data from the Phase II
AAVIATE® trial of ABBV-RGX-314 for the treatment of wet
age-related macular degeneration (wet AMD) using suprachoroidal
delivery. Wet AMD is a chronic, life-long disease with available
anti-VEGF treatment options that may reduce the risk of blindness,
but require frequent injections. Investigational ABBV-RGX-314 using
suprachoroidal delivery is designed to be a one-time, in office
treatment that has the potential to sustain constant anti-VEGF
therapy and stabilize or improve vision long-term for wet AMD
patients. The new data presented at the Hawaiian Eye and Retina
meeting in Maui, HI by
John Pitcher, M.D., Eye Associates
of New Mexico, includes 6-month
results from two additional dose level 3 cohorts (Cohorts 5 and 6).
ABBV-RGX-314, being developed in collaboration with AbbVie, is also
being investigated as a potential one-time gene therapy for the
treatment of diabetic retinopathy and other chronic retinal
conditions.
"The potential of ABBV-RGX-314 to transform the way chronic
retinal conditions are treated has been demonstrated in multiple
trials using both subretinal and suprachoroidal delivery, and these
latest interim results from the AAVIATE trial continue to support
this outlook," said Steve Pakola,
M.D., Chief Medical Officer of REGENXBIO. "One-time, in-office
treatment with ABBV-RGX-314 continues to be well tolerated in
patients with wet AMD. We are pleased that dose level 3 in this
trial has shown the highest reduction in treatment burden with zero
cases of inflammation observed in patients who received
prophylactic short-course topical steroids. We are encouraged by
this data as part of the global ABBV-RGX-314 program."
"Real world evidence shows that patients with wet AMD are often
undertreated due to the unsustainable treatment burden of frequent
injections. As a result, many wet AMD patients experience
significant vision loss over time," said Dr. Pitcher. "I am
encouraged by the safety profile of ABBV-RGX-314 using
suprachoroidal delivery and the potential of a one-time, in-office
injection that can sustain constant anti-VEGF therapy to address a
major unmet need for patients."
Data and Safety Summary
AAVIATE is a Phase II,
multi-center, open-label, randomized, active-controlled,
dose-escalation trial that is evaluating the efficacy, safety and
tolerability of suprachoroidal delivery of
ABBV-RGX-314 in patients with wet AMD. The primary
endpoint of the trial is mean change in vision in patients dosed
with ABBV-RGX-314, as measured by best corrected visual acuity
(BCVA) at Week 40 from baseline, compared to patients
receiving monthly injections of ranibizumab. Other
endpoints include mean change in central retinal thickness (CRT)
and number of anti-vascular endothelial growth factor
(anti-VEGF) intravitreal injections
received following administration of ABBV-RGX-314.
As of November 6, 2023,
ABBV-RGX-314 suprachoroidal delivery was well tolerated across 106
patients from three dose levels. No drug-related SAEs were
reported. All treatment emergent adverse events (TEAEs) through 6
months in the study eye were mild or moderate and included
conjunctival hemorrhage, increased intraocular pressure,
episcleritis, and conjunctival hyperemia. Mild intraocular
inflammation was reported at similar incidence rate in the first
and second dose levels, with mild to moderate intraocular
inflammation seen at the third dose level in Cohort 4 and 5. All
intraocular inflammation resolved with topical corticosteroids.
Notably, there were zero cases of intraocular inflammation in dose
level 3, Cohort 6 (n=21), in which patients received a short-course
of prophylactic topical steroids following administration of
ABBV-RGX-314.
Patients treated with ABBV-RGX-314 continue to demonstrate
stable BCVA and CRT at six months. In addition, a meaningful
reduction in anti-VEGF treatment burden was observed following
administration of ABBV-RGX-314, The highest reduction was seen in
dose level 3, demonstrating an 80% reduction in annualized
injection rate with 50% of patients remaining injection-free.
Data presented is available on the "Publications" section of the
REGENXBIO website at www.regenxbio.com.
Conference Call
In connection with this announcement,
REGENXBIO will host a conference call to discuss the Phase II
AAVIATE trial data with Dr. Pitcher and Allen Ho, M.D., FACS, FASRS, Co-Director, Wills
Eye Retina Service and Director, Retina Research, on Tuesday, January 16 at 4:30 p.m. EST. Listeners can register for the
webcast via this link. Analysts wishing to participate in the
question and answer session should use this link. A replay of
the webcast will be available via the company's investor website
approximately two hours after the call's conclusion. Those who plan
on participating are advised to join 15 minutes prior to the start
time.
About the AAVIATE® Trial
The multi-center,
open-label, randomized, active-controlled, dose-escalation Phase II
AAVIATE trial is evaluating the efficacy, safety and tolerability
of suprachoroidal delivery of ABBV-RGX-314 in patients with wet AMD
using the Clearside SCS Microinjector®. Patients have
been dosed across three dose levels: 2.5x1011 (Cohort
1), 5x1011 (Cohorts 2 and 3), and 1x1012
(Cohorts 4-6) genomic copies per eye (GC/eye). Cohort 5 is
evaluating ABBV-RGX-314 in 20 patients who are NAb positive. Cohort
6 includes a short course of prophylactic steroids following
administration of ABBV-RGX-314.
About ABBV-RGX-314
ABBV-RGX-314, being developed
in collaboration with AbbVie, is being investigated as
a potential one-time treatment for wet AMD, diabetic
retinopathy, and other chronic retinal conditions. ABBV-RGX-314
consists of the NAV® AAV8 vector, which
encodes an antibody fragment designed to inhibit vascular
endothelial growth factor (VEGF). ABBV-RGX-314 is
believed to inhibit the VEGF pathway by which new,
leaky blood vessels grow and contribute to the accumulation of
fluid in the retina.
REGENXBIO is advancing research in two separate routes of
administration of ABBV-RGX-314 to the eye, through a standardized
subretinal delivery procedure as well as delivery to the
suprachoroidal space. REGENXBIO has licensed certain exclusive
rights to the SCS Microinjector® from Clearside
Biomedical, Inc. to deliver gene therapy treatments to the
suprachoroidal space of the eye.
About Wet AMD
Wet AMD is characterized by loss of
vision due to new, leaky blood vessel formation in the retina. Wet
AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with
wet AMD in these geographies alone. Current anti-VEGF therapies
have significantly changed the landscape for treatment of wet AMD,
becoming the standard of care due to their ability to prevent
progression of vision loss in the majority of patients. These
therapies, however, require life-long repeated intraocular
injections to maintain efficacy. Due to the burden of treatment, it
is difficult for patients to adhere to frequent injections, which
can lead to a decline in vision over time.
About REGENXBIO Inc.
REGENXBIO is a leading
clinical-stage biotechnology company seeking to improve lives
through the curative potential of gene therapy. REGENXBIO's NAV
Technology Platform, a proprietary adeno-associated virus (AAV)
gene delivery platform, consists of exclusive rights to more than
100 novel AAV vectors, including AAV7, AAV8 and AAV9. REGENXBIO and
its third-party NAV Technology Platform Licensees are applying the
NAV Technology Platform in the development of a broad pipeline of
candidates, including late-stage and commercial programs, in
multiple therapeutic areas. REGENXBIO is committed to a "5x'25"
strategy to progress five AAV Therapeutics from our internal
pipeline and licensed programs into pivotal-stage or commercial
products by 2025.
Forward-Looking Statements
This press release includes
"forward-looking statements," within the meaning of Section 27A of
the Securities Act of 1933, as amended, and Section 21E of the
Securities Exchange Act of 1934, as amended. These statements
express a belief, expectation or intention and are generally
accompanied by words that convey projected future events or
outcomes such as "believe," "may," "will," "estimate," "continue,"
"anticipate," "assume," "design," "intend," "expect," "could,"
"plan," "potential," "predict," "seek," "should," "would" or by
variations of such words or by similar expressions. The
forward-looking statements include statements relating to, among
other things, REGENXBIO's future operations and clinical trials.
REGENXBIO has based these forward-looking statements on its current
expectations and assumptions and analyses made by REGENXBIO in
light of its experience and its perception of historical trends,
current conditions and expected future developments, as well as
other factors REGENXBIO believes are appropriate under the
circumstances. However, whether actual results and developments
will conform with REGENXBIO's expectations and predictions is
subject to a number of risks and uncertainties, including the
timing of enrollment, commencement and completion and the success
of clinical trials conducted by REGENXBIO, its licensees and its
partners, the timing of commencement and completion and the success
of preclinical studies conducted by REGENXBIO and its development
partners, the timely development and launch of new products, the
ability to obtain and maintain regulatory approval of product
candidates, the ability to obtain and maintain intellectual
property protection for product candidates and technology, trends
and challenges in the business and markets in which REGENXBIO
operates, the size and growth of potential markets for product
candidates and the ability to serve those markets, the rate and
degree of acceptance of product candidates, and other factors, many
of which are beyond the control of REGENXBIO. Refer to the "Risk
Factors" and "Management's Discussion and Analysis of Financial
Condition and Results of Operations" sections of REGENXBIO's Annual
Report on Form 10-K for the year ended December 31, 2022, and comparable "risk factors"
sections of REGENXBIO's Quarterly Reports on Form 10-Q and other
filings, which have been filed with the U.S. Securities and
Exchange Commission (SEC) and are available on the SEC's website at
www.sec.gov. All of the forward-looking statements made in this
press release are expressly qualified by the cautionary statements
contained or referred to herein. The actual results or developments
anticipated may not be realized or, even if substantially realized,
they may not have the expected consequences to or effects on
REGENXBIO or its businesses or operations. Such statements are not
guarantees of future performance and actual results or developments
may differ materially from those projected in the forward-looking
statements. Readers are cautioned not to rely too heavily on the
forward-looking statements contained in this press release. These
forward-looking statements speak only as of the date of this press
release. Except as required by law, REGENXBIO does not undertake
any obligation, and specifically declines any obligation, to update
or revise any forward-looking statements, whether as a result of
new information, future events or otherwise.
SCS Microinjector® is a trademark of Clearside
Biomedical, Inc. All other trademarks referenced herein are
registered trademarks of REGENXBIO.
Contacts:
Dana
Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
Chris Brinzey
ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com
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